Excision BioTherapeutics, a clinical-stage biotechnology company, has recently made headlines with its announcement regarding the publication of pre-clinical data supporting its innovative CRISPR-based gene therapy candidate, EBT-101. This therapy is designed to functionally cure HIV-1. Here’s a detailed summary of the press release:
- The study results showcased the safety, biodistribution, and on-target editing of the simian immunodeficiency virus (SIV) in non-human primates. This data is crucial as it forms the scientific rationale for the ongoing Phase 1/2 clinical evaluation of EBT-101.
- EBT-001, a simian-specific analogue of EBT-101, has been shown to safely remove the simian form of HIV from the genomes of non-human primates. This supports the safety and on-target editing of Excision’s EBT-101 program targeting HIV, which is currently being evaluated in first-in-human Phase 1/2 clinical trials.
Daniel Dornbusch, the Chief Executive Officer of Excision, expressed his enthusiasm about the positive data from the study. He emphasized that a cure for HIV would address a significant unmet need and highlighted the company’s commitment to advancing this breakthrough science to end the HIV pandemic.
Current HIV Treatment:
Currently, individuals with HIV are required to undergo life-long antiretroviral therapy (ART). While ART effectively reduces viral loads and can minimize the risk of HIV transmission, it doesn’t eliminate latent HIV in the body, which can lead to significant medical conditions.
The newly published data complements the efficacy data from previous pre-clinical studies of EBT-101. It demonstrates the excision of integrated proviral SIV DNA in vivo without any detectable off-target effects. The research was conducted by experts at the Lewis Katz School of Medicine at Temple University.
Dr. Kamel Khalili, a CRISPR gene editing of HIV innovator and Co-Founder of Excision, stated that the results highlight the immense potential of EBT-101 in shaping the future of HIV therapeutics. The study involved treating non-human primates with a single intravenous injection of EBT-001 and monitoring them for three to six months. The findings revealed that EBT-001 was well-tolerated at all dose levels, with no evidence of toxicity.
The promising results from this study signify a potential breakthrough in the fight against HIV. The CRISPR-based cure for HIV could significantly enhance the well-being of those living with the virus. Excision BioTherapeutics continues to lead the way in this groundbreaking research, signaling a hopeful future for the HIV community and the potential of gene editing therapies to cure other viral infectious diseases.
For those interested in diving deeper into the research, the full publication can be accessed here.